Miglustat Gen.Orph 100 mg hard capsules

miglustat

Miglustat Gen.Orph contains the active substance miglustat which belongs to a group of medicines that affect metabolism. It is used to treat two conditions:

• Miglustat Gen.Orph is used to treat mild to moderate type 1 Gaucher disease in adults.

In type 1 Gaucher disease, a substance called glucosylceramide is not removed from your body. It starts to build up in certain cells of the body’s immune system. This can result in liver and spleen enlargement, changes in the blood and bone disease.

The usual treatment for type 1 Gaucher disease is enzyme replacement therapy. Miglustat Gen.Orph is only used when a patient is considered unsuitable for treatment with enzyme replacement therapy.

• Miglustat Gen.Orph is also used to treat progressive neurological symptoms in Niemann-Pick type C disease in adults and in children.

If you have Niemann-Pick type C disease, fats such as glycosphingolipids build up in the cells of your brain. This can result in disturbances in neurological functions such as slow eye movements, balance, swallowing, and memory, and in seizures.

Miglustat Gen.Orph works by inhibiting the enzyme called ‘glucosylceramide synthase’ which is responsible for the first step in the synthesis of most glycosphingolipids.

Always take this medicine exactly as your doctor has told you. Check with your doctor or pharmacist if you are not sure.

• For type 1 Gaucher disease: for adults, the usual dose is one capsule (100 mg) three times a day (morning, afternoon and evening). This means a daily maximum of three capsules (300 mg).
• For Niemann-Pick type C disease: For adults and adolescents (over 12 years old), the usual dose is two capsules (200 mg) three times a day (morning, afternoon and evening). This means a daily maximum of six capsules (600 mg).

For children less than 12 years old, your doctor will adjust the dose for Niemann-Pick type C disease.

If you have a problem with your kidneys you may receive a lower starting dose. Your doctor may reduce your dose, e.g., to one capsule (100 mg) once or twice a day, if you suffer from diarrhoea when taking Miglustat Gen.Orph (see section 4). Your doctor will tell you how long your treatment will last.

Miglustat Gen.Orph can be taken with or without food. You should swallow the whole capsule with a glass of water.

If you have any further questions on the use of this medicine, ask your doctor or pharmacist.

Like all medicines, this medicine can cause side effects, although not everybody gets them.

Most serious side effects:

Some patients have had tingling or numbness in the hands and feet (seen commonly). They could be signs of peripheral neuropathy, due to side effects of Miglustat Gen.Orph or they could be due to existing conditions. Your doctor will perform some tests before and during treatment with Miglustat Gen.Orph to assess this (see section 2).

If you do get any of these effects, please seek medical advice from your doctor as soon as possible.

If you get a slight tremor, usually trembling hands, seek medical advice from your doctor as soon as possible. The tremor often disappears without needing to stop the treatment. Sometimes your doctor will need to reduce the dose or stop Miglustat Gen.Orph treatment to stop the tremor.

Very common effects (may affect more than 1 in 10 people)

The most common side effects are diarrhoea, flatulence (wind), abdominal (stomach) pain, weight loss and decreased appetite.

If you do lose some weight when you start treatment with Miglustat Gen.Orph don’t worry. People usually stop losing weight as treatment goes on.

Common effects (may affect up to 1 in 10 people)

Common side effects of treatment include headache, dizziness, paraesthesia (tingling or numbness), abnormal coordination, hypoaesthesia (reduced sensation to touch), dyspepsia (heartburn), nausea (feeling sick), constipation and vomiting, swelling or discomfort in the abdomen (stomach) and thrombocytopenia (reduced levels of blood platelets). The neurological symptoms and thrombocytopenia could be due to the underlying disease.

Other possible side effects are muscular spasms or weakness, fatigue, chills and malaise, depression, difficulty sleeping, forgetfulness and less libido.

Most patients get one or more of these side effects, usually at the start of treatment or at intervals during treatment. Most cases are mild and disappear quite quickly. If any of these side effects cause problems, consult your doctor. He or she may reduce the dose of Miglustat Gen.Orph or recommend other medicines to help control side effects.

Keep this medicine out of the sight and reach of children.

Do not take this medicine after the expiry date which is stated on the carton after ‘EXP’. The expiry date refers to the last day of that month.

This medicine does not require any special storage conditions.

Do not throw away any medicines via wastewater or household waste. Ask your pharmacist how to throw away medicines you no longer use. These measures will help protect the environment.